mRNA-based therapeutics: powerful and versatile tools to combat diseases
S Qin, X Tang, Y Chen, K Chen, N Fan… - Signal transduction and …, 2022 - nature.com
The therapeutic use of messenger RNA (mRNA) has fueled great hope to combat a wide
range of incurable diseases. Recent rapid advances in biotechnology and molecular …
range of incurable diseases. Recent rapid advances in biotechnology and molecular …
The delivery challenge: fulfilling the promise of therapeutic genome editing
Genome editing has the potential to treat an extensive range of incurable monogenic and
complex diseases. In particular, advances in sequence-specific nuclease technologies have …
complex diseases. In particular, advances in sequence-specific nuclease technologies have …
Next-generation stem cells—ushering in a new era of cell-based therapies
EA Kimbrel, R Lanza - Nature Reviews Drug Discovery, 2020 - nature.com
Naturally occurring stem cells isolated from humans have been used therapeutically for
decades. This has primarily involved the transplantation of primary cells such as …
decades. This has primarily involved the transplantation of primary cells such as …
A new class of medicines through DNA editing
MH Porteus - New England Journal of Medicine, 2019 - Mass Medical Soc
Clinical Genome Editing Therapeutic editing of the human genome has long been a holy
grail of genetic medicine. Different approaches are founded on a bedrock of basic laboratory …
grail of genetic medicine. Different approaches are founded on a bedrock of basic laboratory …
Dimethyl sulfoxide: a central player since the dawn of cryobiology, is efficacy balanced by toxicity?
Dimethyl sulfoxide (DMSO) is the cryoprotectant of choice for most animal cell systems since
the early history of cryopreservation. It has been used for decades in many thousands of cell …
the early history of cryopreservation. It has been used for decades in many thousands of cell …
Gene correction for SCID-X1 in long-term hematopoietic stem cells
M Pavel-Dinu, V Wiebking, BT Dejene, W Srifa… - Nature …, 2019 - nature.com
Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an
effective therapy for monogenic diseases of the blood and immune system. Here we …
effective therapy for monogenic diseases of the blood and immune system. Here we …
Hematopoietic stem cell gene therapy: progress and lessons learned
RA Morgan, D Gray, A Lomova, DB Kohn - Cell stem cell, 2017 - cell.com
The use of allogeneic hematopoietic stem cells (HSCs) to treat genetic blood cell diseases
has become a clinical standard but is limited by the availability of suitable matched donors …
has become a clinical standard but is limited by the availability of suitable matched donors …
CRISPR/Cas9-mediated CCR5 ablation in human hematopoietic stem/progenitor cells confers HIV-1 resistance in vivo
Transplantation of hematopoietic stem cells (HSCs) with a naturally occurring CCR5
mutation confers a loss of detectable HIV-1 in the patient, making ablation of the CCR5 gene …
mutation confers a loss of detectable HIV-1 in the patient, making ablation of the CCR5 gene …
[HTML][HTML] Genome editing with mRNA encoding ZFN, TALEN, and Cas9
Genome-editing technologies based on programmable nucleases have significantly
broadened our ability to make precise and direct changes in the genomic DNA of various …
broadened our ability to make precise and direct changes in the genomic DNA of various …
CCR5-edited CD4+ T cells augment HIV-specific immunity to enable post-rebound control of HIV replication
Background We conducted a phase I clinical trial that infused CCR5 gene–edited CD4+ T
cells to determine how these T cells can better enable HIV cure strategies. Methods The aim …
cells to determine how these T cells can better enable HIV cure strategies. Methods The aim …